Target intelligence / Profile preview

KIT proto-oncogene receptor tyrosine kinase mRNA (KIT mRNA)

Target
KIT mRNA
Molecular classification
mRNA, Nucleic acid
01

Overview

KIT mRNA is the messenger RNA transcript of the KIT gene, which encodes the KIT proto-oncogene receptor tyrosine kinase, also known as CD117 or the stem cell factor receptor (SCFR). This Type III receptor tyrosine kinase is essential for the development, survival, and proliferation of several critical cell lineages, including hematopoietic stem cells, mast cells, melanocytes, and the interstitial cells of Cajal in the gastrointestinal tract. In various human cancers, such as gastrointestinal stromal tumors (GIST), systemic mastocytosis, and acute myeloid leukemia (AML), activating mutations in the KIT gene lead to constitutive, ligand-independent signaling that drives oncogenesis. While small-molecule tyrosine kinase inhibitors (TKIs) like imatinib and avapritinib are standard treatments that target the KIT protein, clinical resistance frequently develops through secondary mutations in the kinase domain. Targeting the KIT mRNA transcript using RNA-based therapeutics, such as small interfering RNAs (siRNAs), antisense oligonucleotides (ASOs), or exon-skipping oligonucleotides (ESOs), offers a strategy to silence the gene and reduce the total pool of oncogenic protein regardless of the specific mutation, potentially overcoming TKI resistance.

Other names
c-Kit mRNACD117 mRNAStem cell factor receptor mRNASCFR mRNAMast/stem cell growth factor receptor mRNAPBT mRNAV-kit Hardy-Zuckerman 4 feline sarcoma viral oncogene homolog mRNA
02

Mechanism of action

RNA interference (RNAi) mediated by siRNA, antisense-mediated mRNA degradation (RNase H-dependent), or steric blocking of translation and splicing modulation (exon skipping).

03

Biological functions

TranslationProtein synthesisRegulation of signal transductionHematopoiesis regulationMelanogenesis regulationGametogenesis regulation
04

Disease associations

Gastrointestinal stromal tumor (GIST)Systemic mastocytosisAcute myeloid leukemia (AML)MelanomaPiebaldismTesticular seminoma
05

Safety considerations

Hematological toxicity (anemia, neutropenia) due to KIT's essential role in hematopoiesisPigmentation disorders (e.g., vitiligo-like patches)Off-target effects of oligonucleotide sequencesDelivery challenges to solid tumor tissuesPotential for innate immune activation by synthetic RNA
06

Interacting drugs

Experimental KIT-targeting siRNAs

2 more in the full profile.

07

Biomarkers

KIT mutation status (e.g., D816V, Exon 11, Exon 9)KIT mRNA expression levelsCD117 protein expression (IHC)Serum tryptase levels (in mastocytosis)

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